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Latest advances in gene therapy for ocular disease are highlighted in Human Gene Therapy
Date:5/9/2011

the rate of retinal degeneration in "AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa."

In the Brief Report "Gene Therapy in Ophthalmology: Validation on Cultured Retinal Cells and Explants from Postmortem Human Eyes," Fradot and colleagues show how gene therapy strategies can be developed and refined using cultured cells and explants from human eyes harvested after death to limit research studies done in primates.

"The most dramatic clinical success of in vivo gene therapy has been realized using AAV vectors in the treatment of a rare inherited form of congenital blindness; some level of functional improvement was realized in three independent trials. A number of scientists from around the world are progressing in vivo gene therapy into a number of other ocular disorders. This issue of the Journal captures some of these exiting new directions," says James M. Wilson, M.D., Ph.D., Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia.


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Contact: Vicki Cohn
vcohn@liebertpub.com
914-740-2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

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